The Rare Moments

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Trinity Life Sciences Trinity Life Sciences Trinity Life Sciences

With extensive commercialization expertise and deep knowledge of rare diseases, Trinity Life Sciences has supported the launches of some of the most impactful drugs and therapies globally. We remain committed to helping our clients overcome unique commercialization challenges faced by specialized treatments and strive to create a better future for those living with rare diseases.

Derek Ansel Admin Derek Ansel Worldwide

Jim Geraghty said it best today at the 9th Annual Rare Disease Day Event at the Broad Institute. “It’s a marathon, not a sprint,” when it comes to rare disease research. Always a science-focused and inspiring event at the Broad!

Katherine StilI Katherine StilI Phelan-McDermid Syndrome Foundation

The Phelan-McDermid Syndrome Foundation is excited to celebrate Rare Disease Day 2024! You can learn more about Phelan-McDermid Syndrome by reviewing our infographic and by visiting our website at pmsf.org.

Infographic

PMSF.org
Aimei Lund Aimei Lund Uplifting Athletes

Yesterday, 2/27, Uplifting Athletes was able to host four rare families at an Uplifting Experience with the Philadelphia Flyers. Families were provided with parking, great seats, and swag bags. Bonus the Flyers got the win!

Canary Advisors Canary Advisors Canary Advisors

Canary Advisors 3

www.linkedin.com/posts/canaryadvisorsllc_raredc2024-rarediseaseday-activity-7163517250669367297-toob?utm_source=share&utm_medium=member_desktop
The N=1 Collaborative The N=1 Collaborative

Connecting, Collaborating, Communicating, Challenges: Sharing experiences navigating family conversations in an n-of-1 investigational treatment setting. Learn more, below!

www.n1collaborative.org/post/connecting-collaborating-communicating-challenges-sharing-experiences-navigating-family-conversa
Amy Raymond Amy Raymond Worldwide Clinical Trials

With approval of the first treatment to use gene editing to address a genetic rare disease, plus all the very recent innovations in base editing, prime editing, and lipid nanoparticle delivery, I’m hopeful we’ll see an ever-increasing number of gene editing programs in development for patient communities with genetically driven rare diseases. Kudos to the pioneers with development programs for rare cardiovascular diseases, rare blood cancers, rare hemoglobinopathies, rare bleeding disorders, rare metabolic disorders, rare neuromuscular diseases, and rare inherited retinal diseases!

Canary Advisors Canary Advisors Canary Advisors

Canary Advisors 1

www.linkedin.com/posts/canaryadvisorsllc_raredc2024-rarediseaseday-activity-7166042878010462208-66EC?utm_source=share&utm_medium=member_desktop
Castleman Disease Collaborative Network Castleman Disease Collaborative Network Castleman Disease Collaborative Network

The Castleman Disease Collaborative Network (CDCN) is a global nonprofit organization dedicated to accelerating research and treatment for this rare and often deadly disease, supporting patients on their journeys, and revolutionizing biomedical research to cure countless other diseases. As we recognize Rare Disease Day on February 29, 2024, we are proud to share with you the story of one of the many thousands of Castleman disease patients who inspire our work every day. Learn more at cdcn.org.

Derek Ansel Admin Derek Ansel Worldwide

So incredibly touched to receive this book from our friends at Ultragenyx. Dr. Emil Kakkis has been at the forefront of rare disease research throughout his entire career. I’m looking forward to reading this book throughout Rare Disease Week!

Saving Ryan

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We live for the rare moments.